The FACTs trial for Fabry disease highlights the promise and challenges of gene therapy

Jeffrey A. Medin; Michael L. West

doi:10.1002/ctd2.70028

Clinical and Translational Discovery ›› 2025, Vol. 5 ›› Issue (1) : e70028 DOI: 10.1002/ctd2.70028

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The FACTs trial for Fabry disease highlights the promise and challenges of gene therapy

Jeffrey A. Medin ¹^,²^,^†
, Michael L. West ³^,^†

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Jeffrey A. Medin, Michael L. West. The FACTs trial for Fabry disease highlights the promise and challenges of gene therapy. Clinical and Translational Discovery, 2025, 5(1): e70028 DOI:10.1002/ctd2.70028

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References

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[1]	KhanA, BarberDL, McKillopWM, et al. Lentivirus-mediated gene therapy for Fabry disease:5-year end-of-study results from the Canadian FACTs trial. Clin Transl Med. 2024:e70073.

[2]	KhanA, BarberDL, HuangJ, et al. Lentivirus-mediated gene therapy for Fabry disease. Nat Comm. 2021;12:1178.

[3]	SalehAH, RotheM, BarberDL, et al. Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease. Mol Ther Methods Clin Dev. 2023;28:262-271.

[4]	TucciF, Galimberti S, NaldiniL, et al. A systemic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders. Nat Comm. 2022;13:1315.

[5]	DuncanCN, Bledsoe JR, GrzywaczB, et al. Hematologic cancer after gene therapy for cerebral adrenoleukodystrophy. N Engl J Med. 2024;391:1287-1301.

[6]	HuangJ, KhanA, AuBC, et al. Lentivector iterations and pre-clinical scale-up/toxicity testing: targeting mobilized CD34+ cells for correction of Fabry disease. Mol Ther Methods Clin Dev. 2017;5:241-258.

[7]	NagreeMS, Felizardo TC, FaberML, et al. Autologous, lentivirus-modified, T-rapa cell ‘micropharmacies’ for lysosomal storage disorders. EMBO Mol Med. 2022;14(4):e14297.

[8]	AssafB. Systemic toxicity of recombinant adeno-associated virus gene therapy vectors. Toxicol Pathol. 2024;1926233241298892.

[9]	BraunF, AbedA, SellungD, et al. Synuclein alpha accumulation mediates podocyte injury in Fabry nephropathy. J Clin Invest. 2023;133(11):e157782.

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2024 The Author(s). Clinical and Translational Discovery published by John Wiley & Sons Australia, Ltd on behalf of Shanghai Institute of Clinical Bioinformatics.